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Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII

Enzyme replacement therapy (ERT) effectively reverses storage in several lysosomal storage diseases. However, improvement in brain is limited by the blood-brain barrier except in the newborn period. In this study, we asked whether this barrier could be overcome by higher doses of enzyme than are use...

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Detalhes bibliográficos
Main Authors: Vogler, Carole, Levy, Beth, Grubb, Jeffrey H., Galvin, Nancy, Tan, Yun, Kakkis, Emil, Pavloff, Nadine, Sly, William S.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2005
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC1253584/
https://ncbi.nlm.nih.gov/pubmed/16162667
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0506892102
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